ARGININE SUPPLEMENTATION IN SICKLE CELL ANEMIA-PHYSIOLOGICAL AND PROPHYLATIC

本子项目是利用由NIH/NCRR资助的中心赠款提供的资源的众多研究子项目之一。子项目和研究者（PI）可能已经从另一个NIH来源获得了主要资金，因此可以在其他CRISP条目中表示。列出的机构是中心的，不一定是研究者的机构。镰状细胞病是一种严重的遗传性红细胞疾病，可导致严重贫血和血管闭塞发作。这种血管阻塞影响到身体的每个器官系统，并导致反复发作的疼痛，压倒性的感染和进行性器官损伤。一氧化氮是一种由精氨酸产生的物质，在镰状细胞病的血管阻塞中具有许多有利的功能。精氨酸是一种氨基酸，已被证明可以提高一氧化氮，并对镰状细胞病患者有有益的结果，这将有助于确定每日精氨酸是否是镰状细胞病患者的有益研究药物。该研究是一项双盲、安慰剂对照试验，符合条件的镰状细胞病患者（儿童、青少年和成人）将在12周内口服精氨酸或安慰剂。受试者将随机接受安慰剂或两种剂量精氨酸（0.04g/kg/天）中的一种。

This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. Sickle cell disease is a serious inherited disease of the red blood cells resultiing in sever anemia and episodes of occlusion of the blood vessels. This vaso-occlusion affects every organ system in the body and results in recurrent episodes of pain, overwhelming infections and progressive organ damage. Nitric oxide is a substance produced by arginine and has many functions that could impact favorably in vaso-occlusion in sickle cell disease. Arginine is an aminoacid that has been shown to raise nitric oxide and have benificial results in subjects with sickle cell disease, and will help to determine if daily arginine is a benificial investigational drug for subjects with sickle cell disease. This study is a double-blind, placebo-controlled trial in which eligible subjects (children, adolescents, and adults) with sickle cell disease will receive either arginine or placebo by mouth during 12 weeks. Subjects will be randomized to recieve placebo or one of two doses of arginine (0.04g/kg/day).